Improving the production and release of gene therapy vectors
Official title: Engineering the biology of AAV secretion and production
Funded at Duke University · NIH-11112373 · Data updated 2026-11-12
This study is looking at ways to make more of a helpful virus used in gene therapy, which could lead to better and safer treatments for various diseases, making them easier for patients to access.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Duke University NIH-funded |
| Lab location | 1 site (Durham, United States) |
| Project start | May 1, 2023 |
| Project end | April 30, 2027 |
| Funding year | 2025 |
| Award (this year) | $456,997 |
| NIH project number | 5R01EB034573-03 |
| Project ID | NIH-11112373 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are patients with genetic disorders that could be treated with AAV-based gene therapies.
✕Less likely to help
Patients with conditions that are not addressed by AAV-based therapies may not benefit from this research.
What this research studies
This research focuses on enhancing the production and secretion of adeno-associated viruses (AAV), which are important tools in gene therapy for various diseases. By investigating how AAV exits host cells, the researchers aim to optimize the manufacturing process, potentially leading to higher yields and better quality of these viral vectors. This could improve the effectiveness of gene therapies, making them safer and more accessible for patients. The study employs advanced biological techniques to understand the mechanisms behind AAV secretion and its implications for therapy.
Why it matters
Potential benefit: If successful, this research could lead to more effective and safer gene therapies for a range of diseases.
How similar studies have gone: Previous research has shown promise in optimizing AAV production, indicating that this approach could lead to significant advancements in gene therapy.
Where this research is happening
-
Duke University
Durham, United States Active
Researchers
- Principal investigator: Asokan, Aravind — Duke University
- Study coordinator: Asokan, Aravind
About this research
- This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
- Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
- For full project details, budget, and progress reports, visit the official NIH RePORTER page.